A French drugmaker that has licensed a treatment for rare genetic muscular disorders from a US firm plans to use money raised in a Series A funding round to conduct a clinical trial starting next year.

Strasbourg, France-based Dynacure said Friday that it had completed the Series A round, worth €47 million ($55 million), led by Andera Partners, with participation by Pontifax, Bpifrance, Kurma Partners and IdInvest Partners. The drug, Dyn101, was in-licensed from Carlsbad, California-based Ionis Pharmaceuticals, which develops antisense drugs targeting RNA molecules. Dynacure is developing the drug in collaboration with Ionis for centronuclear myopathy, or CNM, a group of disorders causing muscle weakness that can require use of wheelchairs, ventilators and gastric tubes. Dyn101 targets the most common X-linked form of CNM and the autosomal dominant form. Ionis is a minority shareholder in Dynacure, CEO Stephane van Rooijen said in a phone interview.

The company finalized its selection of the drug for clinical development in 2017 and plans to enter it into a clinical trial next year, van Rooijen said. The Phase I study will take place at Europe only, but the company would subsequently conduct a potentially registration-directed Phase II/III study that would include US sites.

The sites for the Phase I study have not yet been confirmed, but patients will be recruited through expert centers across Europe, van Rooijen said. The trial would also tap patient organizations. Recruitment is always a challenge in rare diseases, making it necessary to work with centers to help that happen, he said, adding that the trial would have fewer than 30 patients.

ClinicalTrials.gov lists only 10 studies in CNM, most of them focused on natural history, genetic testing and the like, rather than on new drugs. In May, San Francisco-based Audentes Therapeutics announced “positive” early data from the Phase I/II ASPIRO trial of AT132 in patients with X-linked myotubular myopathy, one of the CNM subtypes, including “significant” improvements in neuromuscular and respiratory function at the 24-week timepoint. AT132 is an adeno-associated virus-based gene therapy that contains a functional copy of the MTM1 gene, mutations in which cause XLMTM.

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